FDA Approves Omlonti (omidenepag isopropyl ophthalmic solution) for Reduction of Elevated Intraocular Pressure in Primary Open-Angle Glaucoma or Ocular Hypertension

Santen Inc., the U.S. subsidiary of Santen Pharmaceutical Co., Ltd. (Santen), and UBE Corporation (UBE)   announced   the U.S. Food and Drug Administration (FDA)   approval of Omlonti (omidenepag isopropyl ophthalmic solution) 0.002% eye drops for the reduction of elevated intraocular pressure (IOP) in patients with primary open-angle glaucoma or ocular hypertension. The approval date was September 22.

Omlonti is developed jointly by Santen and UBE. Omidenepag isopropyl, the active pharmaceutical ingredient in Omlonti, developed by UBE, is a relatively selective prostaglandin EP2 receptor agonist, which increases aqueous humor drainage through the conventional (or trabecular) and uveoscleral outflow pathways, and the only product with this pharmacological action. Omlonti was launched in Japan as Eybelis® ophthalmic solution 0.002% in November 2018, and was filed for marketing approval in Asian countries in stages. The product was released in five countries and regions beginning in February 2021.

“Glaucoma prevalence is increasing as the global population ages. Supporting patients by protecting vision across the continuum of clinical care in glaucoma is a significant aim for Santen to reduce the social and economic opportunity loss of people around the world caused by eye conditions,” explains Peter Sallstig, Chief Medical Officer of Santen. “This approval is an important milestone in our ambition to tackle unmet needs in eye health and advances our goal of realization of “Happiness with Vision”. It also represents our first glaucoma offering in the U.S. We are pleased to provide doctors and patients in the U.S. with a new option to help control IOP for the more than three million Americans affected by glaucoma1 or ocular hypertension.”

“UBE Corporation is committed to working on new drug discoveries on a daily basis with the aim of providing patients with more treatment options for diseases with high unmet needs," said Yoichi Funayama, Senior Executive Officer and General Manager of the Pharmaceutical Division, UBE Corporation. “We are very pleased that this ophthalmic solution has been approved for glaucoma in the U.S., following approvals in Japan and Asia. We have high expectations that omidenepag isopropyl will provide a new treatment option for more patients suffering from glaucoma and ocular hypertension through Santen.”

Omlonti was evaluated in three randomized and controlled clinical trials in subjects with open-angle glaucoma or ocular hypertension with average baseline IOP of 24-26 mm Hg. The double-masked treatment duration was three months in all three studies. The third study included a 9-month open-label treatment period following the 3-month double-masked treatment period. In the three studies, IOP reductions were observed for all treatment arms. In the Omlonti arm, the reduction in IOP ranged from 5-7 mm Hg across all three studies. The corresponding reductions for the timolol and latanoprost arms were 5-7 mm Hg and 6-8 mm Hg, respectively.

Glaucoma causes damage to the optic nerve resulting in visual field loss, and remains a leading cause of irreversible blindness worldwide.2 Since the disease is generally progressive, early detection and treatment to control the progression are crucial, and lowering IOP is the most effective means of avoiding damage to the optic nerve. The estimated number of patients globally in 2020 was 76 million, and it is expected to increase to 95 million by 2030.3 Primary open-angle glaucoma is the most common type of glaucoma. Ocular hypertension, which affects millions, can lead to glaucoma and vision loss if untreated.4

“Treatments that focus on IOP reduction help to slow or prevent further loss of vision for those with glaucoma or ocular hypertension. However, not all patients respond to the same treatments, and some may not have successful outcomes,” said Jason Bacharach, MD, Medical and Research Director at North Bay Eye Associates, Inc. “The approval of omidenepag isopropyl ophthalmic solution 0.002% provides doctors with another safe and effective option to use when treating patients with these sight-threatening conditions.”

 https://en.wikipedia.org/wiki/Omidenepag

FDA Approves Iheezo (chloroprocaine hydrochloride ophthalmic gel) for Ocular Surface Anesthesia

Harrow (Nasdaq: HROW), an eyecare pharmaceutical company exclusively focused on the discovery, development, and commercialization of innovative ophthalmic therapies, and Sintetica, S.A., a growing pharmaceutical company focused on analgesics, local anesthetics, and sterile injectable solutions,  announced the U.S. Food and Drug Administration (FDA) approval of Iheezo (chloroprocaine hydrochloride ophthalmic gel) 3% for ocular surface anesthesia. Iheezo is a sterile, single-patient‑use, physician‑administered, ophthalmic gel preparation, containing no preservatives, that is safe and effective for ocular surface anesthesia. Iheezo represents the first approved use in the U.S. ophthalmic market of chloroprocaine hydrochloride and the first branded ocular anesthetic approved for the U.S. ophthalmic market in nearly 14 years. Iheezo is protected by an Orange Book-listed patent that is valid until 2038.

“On behalf of all our ophthalmic physician partners and the patients they serve, we and our partners at Sintetica are grateful to the FDA for a New Drug Application (NDA) review process that resulted in the approval of Iheezo in advance of our PDUFA target action date,” said Mark L. Baum, Harrow Chairman and Chief Executive Officer. “We have always believed in the unique clinical value of Iheezo, and now that Iheezo is approved for use in the U.S. market, it has the potential to become an indispensable premium tool for eyecare professionals and their patients requiring ocular surface anesthesia.”

Nicola Caronzolo, Sintetica Chief Executive Officer, added, “I am particularly proud of this important milestone, which exemplifies the quality of Sintetica’s research and development groups and our ability to innovate – to be a global pharmaceuticals leader. I want to give special thanks to our regulatory group, who while working with the Harrow team, performed extraordinarily well, resulting in this early U.S. market approval for this important new medicine.”

The safety and efficacy of Iheezo were demonstrated in three human clinical studies. Studies 1 and 2 were randomized, double-blinded, placebo-controlled studies that evaluated the effect of Iheezo on healthy volunteers, and Study 3 was a randomized, prospective, multi-center, active-controlled, observer‑masked study that evaluated the administration of Iheezo in patients undergoing cataract surgery. Study 3 marks the first time a U.S. drug candidate was studied in a surgical model for FDA approval in the ocular surface anesthesia category. This study demonstrated that Iheezo not only worked rapidly (about 1 to 1.5 minutes) and provided sufficient anesthesia to successfully perform the surgical procedure (on average lasting 22 minutes), but importantly, no patient dosed with Iheezo required a supplemental treatment to complete the surgical procedure.

According to a September 2021 report by Market Scope, there are an estimated 4.5 million cataract surgeries and over 8 million intravitreal injections performed annually in the U.S., all of which typically utilize some form of ocular surface anesthesia.

Baum continued, “Harrow currently provides perioperative medications for a significant number of the U.S. ophthalmic surgical procedures. We believe our customer base of more than 10,000 ophthalmologists, optometrists, retina specialists, outpatient hospital facilities, and ambulatory surgery centers will appreciate the unique clinical value and practice efficiency Iheezo offers, including its single‑use packaging format, which according to the Institute for Safe Medication Practices (ISMP), decreases the risk of infection and medication errors associated with the use of communal eye drops.

“We have been planning for the commercial launch of Iheezo for over a year, and with our national market access and sales organization already in place, we are 100% ready. Given our earlier FDA approval date, we have accelerated our market access strategy to support a commercial launch date slightly ahead of our previously planned launch at the May 2023 American Society of Cataract and Refractive Surgery (ASCRS) meeting in San Diego, CA.”

https://en.wikipedia.org/wiki/Chloroprocaine

FDA Approves Iheezo (chloroprocaine hydrochloride ophthalmic gel) for Ocular Surface Anesthesia

FDA Approves Relyvrio (sodium phenylbutyrate/taurursodiol) for Patients with Amyotrophic Lateral Sclerosis (ALS)

In continuation of my update on phenyl butyrate and taurursodiol..

Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”)   announced  the U.S. Food and Drug Administration (FDA)   approval of Relyvrio (sodium phenylbutyrate and taurursodiol) for the treatment of adults with amyotrophic lateral sclerosis (ALS). Relyvrio (previously known as AMX0035 in the U.S.) significantly slowed the loss of physical function in people living with ALS in a randomized, placebo-controlled clinical trial. Relyvrio can be taken as a monotherapy or with existing approved treatments.

“Today’s FDA approval of Relyvrio is an exciting milestone for the ALS community and is a major step toward achieving our mission to one day end the suffering caused by neurodegenerative diseases,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. “We want to give a heartfelt thank you to the broader ALS community, including healthcare professionals and those living with ALS, for their guidance, support of our clinical programs, and for sharing their experiences with us. Their stories inspired us and helped our team to better understand the ALS clock, instilling in us a deep sense of urgency that will continue to drive us forward. This is just the beginning and there is much more to be done.”
ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis and eventually, death. More than 90% of people with ALS have sporadic disease, showing no clear family history. ALS affects approximately 29,000 people in the U.S.

Leading U.S. ALS advocacy organizations including The ALS Association, Answer ALS Foundation, I AM ALS, Les Turner ALS Foundation and Team Gleason said in a statement, “Our organizations have been on a mission to create a world free of ALS. With today’s approval, we are encouraged that Relyvrio can offer people living with ALS and their families the potential of more time with functional independence. This is especially important for a rapidly progressive disease with a median survival time from diagnosis of just two to three years. This is significant for people living with ALS, their loved ones, caregivers, clinicians, researchers, and advocacy, as we now have a new treatment option that could be a big step forward for the future of ALS care.”

The approval of Relyvrio is based on data from CENTAUR, a multicenter Phase 2 clinical trial in 137 participants with ALS encompassing a 6-month randomized, placebo-controlled phase and an open-label extension (OLE) long-term follow-up phase. Detailed data from CENTAUR were published in the New England Journal of Medicine, Muscle & Nerve, and the Journal of Neurology, Neurosurgery, and Psychiatry.

The most common adverse events occurring with Relyvrio (at least 15% and at least 5% greater than placebo) were diarrhea, abdominal pain, nausea, and upper respiratory tract infection. Gastrointestinal-related adverse reactions occurred throughout the study but were more frequent during the first three weeks of treatment.

“Any time we have a new tool to slow the progression of this disease represents an important milestone in how we battle ALS. The published data on both function and survival in a randomized trial – and what this means for people living with ALS – are a step forward for the ALS community,” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, and Associate Professor of Physical Medicine and Rehabilitation at Harvard Medical School and Spaulding Rehabilitation Hospital.

FDA Approves Relyvrio (sodium phenylbutyrate/taurursodiol) for Patients with Amyotrophic Lateral Sclerosis (ALS)

FDA Approves Lytgobi (futibatinib) for Previously Treated, Unresectable, Locally Advanced or Metastatic Intrahepatic Cholangiocarcinoma

Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd. announced today that the U.S. Food and Drug Administration (FDA) has approved Lytgobi tablets for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma (iCCA) harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.

This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

“Lytgobi is an effective, well-tolerated therapy for patients with intrahepatic CCA that can be taken orally,” said Tim Whitten, President and CEO of Taiho Oncology, Inc. “This approval is an important milestone for patients and may provide hope for improved outcomes. As someone whose family has been impacted by cholangiocarcinoma, I’m acutely aware of the impact this disease can have on the patient and their loved ones.”

As a whole, cholangiocarcinoma is an aggressive cancer of the bile ducts and is diagnosed in approximately 8,000 individuals each year in the U.S.1 This includes both intrahepatic (inside the liver) and extrahepatic (outside the liver) forms of the disease. Approximately 20% of patients diagnosed with CCA have the intrahepatic form of the disease.2,3 Within this 20%, approximately 10-16% of patients have FGFR2 gene rearrangements, including fusions, which promote tumor proliferation.4,5,6,7,8 Lytgobi covalently binds to FGFR2 and inhibits the signaling pathway.9 The other approved FGFR inhibitors are reversible ATP-competitive inhibitors.10,11,12

“Lytgobi is a key example of the potential of precision medicine in iCCA and represents another advance in the treatment of this rare and challenging disease,” said medical oncologist Lipika Goyal, MD, MPhil, of the Massachusetts General Hospital Cancer Center and lead investigator of the pivotal study that led to the approval of Lytgobi. “I am encouraged that treatment options continue to expand and evolve for this disease through the dedicated efforts of many over several years.”

The approval of Lytgobi is based on the results of the primary analysis of the FOENIX*-CCA2 trial, a global Phase 2 open-label trial evaluating 103 patients with unresectable, locally advanced or metastatic iCCA harboring FGFR2 gene rearrangements including fusions. In this trial, patients received Lytgobi orally once daily at a dose of 20mg until disease progression or unacceptable toxicity.

The trial met its primary endpoint with an objective response rate of 42% as measured by independent central review. The median duration of response (DOR) was 9.7 months, with 72% of responses lasting at least six months. The most common (≥20%) adverse reactions were nail toxicity, musculoskeletal pain, constipation, diarrhea, fatigue, dry mouth, alopecia, stomatitis, dry skin, arthralgia, dysgeusia, abdominal pain, dry eye, nausea, decreased appetite, urinary tract infection, palmar-plantar erythrodysesthesia syndrome, and vomiting.

Lytgobi was discovered by Taiho Oncology’s parent company, Taiho Pharmaceutical, which continues to co-develop this product for other potential tumor types. “The Taiho group is working as one to optimize this agent for the patients who are waiting,” said Teruhiro Utsugi, Senior Managing Director at Taiho Pharmaceutical.

https://en.wikipedia.org/wiki/Futibatinib

FDA Approves Furoscix (furosemide injection) for the At-Home Treatment of Congestion Due to Fluid Overload in Chronic Heart Failure

scPharmaceuticals Inc. (Nasdaq: SCPH), a pharmaceutical company focused on developing and commercializing products that have the potential to optimize the delivery of infused therapies, advance patient care, and reduce healthcare costs,  announced   the U.S. Food and Drug Administration (FDA)   approval of  Furoscix (furosemide injection), a proprietary formulation of furosemide delivered via an On-Body Infusor for the treatment of congestion due to fluid overload in adults with New York Heart Association Class II/III chronic heart failure. Furoscix is not indicated for emergency situations or in patients with acute pulmonary edema. Furoscix Infusor will deliver only an 80-mg dose. Furoscix is the first and only FDA-approved subcutaneous loop diuretic that delivers IV equivalent diuresis at home via the Furoscix Infusor.

“Congestion due to worsening heart failure is one of the most common causes of hospital admissions in patients over 65, and today’s approval of Furoscix represents an important treatment advancement for the over seven million heart failure patients in the U.S. that will be able to self-administer IV equivalent diuresis at home,” said John Tucker, President and Chief Executive Officer of scPharmaceuticals. “We are preparing to optimize commercialization efforts to offer Furoscix to patients in the first quarter of next year with the goal of driving rapid patient adoption to meet the needs of the $5.9 billion addressable market in the U.S.”

IV equivalence was established in a clinical study in which Furoscix demonstrated 99.6% bioavailability (90% CI: 94.8%-104.8%) and 8-hour urine output of 2.7 L which was similar to subjects receiving intravenous furosemide. Furoscix is not indicated for use in emergency situations or in patients with acute pulmonary edema. The On-Body Infusor will deliver only an 80-mg dose of Furoscix.

“As we move towards commercialization, we have compiled a body of evidence demonstrating the value proposition of Furoscix across healthcare stakeholders,” said John Mohr, Pharm.D., Senior Vice President, Clinical Development and Medical Affairs of scPharmaceuticals. “The totality of clinical and pharmacoeconomic data that we have generated to date supports an opportunity to shift the treatment paradigm of how heart failure patients with congestion are treated and has the potential to become a new standard of care.”

Furoscix enables subcutaneous administration at home by the patient or a caregiver with the use of the Furoscix On-Body Infusor. The On-Body Infusor for Furoscix was developed utilizing West Pharmaceutical Services’ proprietary SmartDose®1 On-Body Drug Delivery technology. Once the pre-filled cartridge is inserted into the pre-programmed single-use On-Body Infusor for Furoscix and attached to the abdomen, the device is activated with the press of a button to deliver an 80-mg dose over five hours.

“This marks a tremendous opportunity to improve the at-home management of worsening congestion in patients with heart failure who display reduced responsiveness to oral diuretics and require administration of intravenous diuretics, which typically requires admission to the hospital,” said William T. Abraham, M.D., Professor of Internal Medicine (Cardiology), Physiology and Cell Biology and College of Medicine Distinguished Professor at The Ohio State University and scPharmaceuticals Board member. “The FDA’s approval of Furoscix is significant and will allow patients to be treated outside of the hospital setting, and I look forward to incorporating it into my own practice as quickly as possible.”

Furoscix® is indicated for the treatment of congestion due to fluid overload in adult patients with New York Heart Association (NYHA) Class II and Class III chronic heart failure.

Furoscix is not indicated for use in emergency situations or in patients with acute pulmonary edema. The On-Body Infusor will deliver only an 80-mg dose of Furoscix.

More...

https://en.wikipedia.org/wiki/Furosemide